Urgent provincial/territorial action needed to address the crisis in rare disease in Canada

VIA EMAIL TO CANADA’S PROVINCIAL AND TERRITORIAL HEALTH MINISTERS  

November 1, 2022 
 
Subject: Urgent provincial/territorial action needed to address the crisis in rare disease in Canada 

Dear Provincial and Territorial Ministers of Health, 

Please put Rare Disease at the very TOP of your agenda as you convene for your annual Health Ministers meeting. I believe you will understand why upon reading this letter. 

As all health ministries struggle with the impact that COVID had on healthcare resources, it may be tempting to put rare diseases on the back burner… again… until other issues are resolved. That would be a costly mistake. 

About 3.2 million Canadians have a rare disease, more than the number with diabetes, cardiovascular disease or all cancers combined. More than two-thirds affected are children. More than a third of those children will die before their fifth birthday. In Canada, every 39 minutes, a child dies of a rare disease.  

The data from developed countries across Europe, the UK, USA, and Australia, as well as our own Canadian data, demonstrate the inordinate impact of rare disease on health resources, a crisis that has only worsened post-COVID.  

• In the USA, genetic (mostly rare) diseases account for up to approximately 50% of paediatric hospital inpatient costs 
• In the USA, just a subset of <379 of the 7,000 RD, accounted for nearly $US966M direct and indirect cost in 2019 
• This cost is greater than many other common conditions, e.g., cancer, diabetes, heart disease and stroke, Alzheimer’s dementia and arthritis.
•  Two new studies in 2021 confirm these figures and also demonstrate that nationally RD account for 1.5 times ($US 105 Billion) the paediatric inpatient costs compared to all common diseases ($US 150 Billion versus $US70 Billion), and have a proportionately greater impact on children and youth versus adult costs 
• In Ireland, 6 in 10 deaths in children and 9 in 10 hospital bed days for death are attributed to RD 
• In Canada, 60% or more of ER admissions in major pediatric hospitals are children with rare conditions, some diagnosed and some undiagnosed 

The previous patient survey conducted by the Canadian Organization for Rare Disorders documented the long delay to diagnosis (up to seven years), the numerous misdiagnoses resulting in missed opportunities to prevent serious symptoms or death as well as delayed or harmful wrong treatments. We expect that our new survey which is currently in the field will confirm these sobering statistics, because little has changed in how we treat rare disease. 

Clearly, doing nothing to address the rare disease crisis is costly … in terms of dollars and lives.  

You may know that the Chinese word for crisis is made up of two words: danger and opportunity. Luckily, we have the opportunity RIGHT NOW to do something EXTRAORDINARY. 

As you know, in 2019, the federal government committed $1 billion to set up a national Rare Disease Drug Strategy, with an additional $500 million annually. After 3 years of CORD-led multi-stakeholder discussions, there appears to be broad agreement that optimal benefits of a rare drug program can only be achieved if we have a rare disease infrastructure, built on a national network of Rare Disease Centres of Excellence. When fully implemented, this “hub and spoke” network would assure that rare disease patients will get timely and accurate diagnosis, referral to the right specialist, a personal care plan managed by healthcare professionals close to home, and active participation in on-going monitoring.   

The second and related major issue is a modern Rare Disease Drug Strategy that is “fit for purpose” of assuring timely access to innovative medicines. Given that only 5% of rare diseases have an approved therapy, CORD calls for investment in academic and non-profit R&D, incentives to encourage commercially developed therapies and sustainable financing models for both types of therapies to assure that Canadian patients have access as soon as possible.  

The crisis in rare disease constitutes a clear and present danger not only to the patients and families affected but to the whole healthcare system and to society. But there is an unprecedented opportunity to invest wisely the $1 billion start-up fund and the $500 million annual federal contribution to establishing a “state of the art” Rare Disease Strategy to support a Rare Disease Drug Strategy that optimizes benefits to patients and their families and to all of society. 

We urge the provincial and territorial Ministers of Health to ACT NOW to adopt a consensual approach in meeting with the federal Minister of Health to accept and invest smartly the $500 million annual allocation for rare diseases. 

Over 3 million Canadians are counting on you and we know you will not let us down. 

MAKE A RARE DISEASE STRATEGY YOUR HEALTHCARE LEGACY TO CANADIANS!!! 

Sincerely, 

Durhane Wong-Rieger 
President & CEO 
Canadian Organization for Rare Disorders 
151 Bloor Street West, Suite 600 
Toronto, Ontario M5S 1S4 

Encl.      CORD Proposal for Canada’s Rare Disease Drug Program within a Canadian Rare Disease System, including Rare Disease Centres of Excellence